Recombinant human growth hormone for treatment of Turner Syndrome: systematic review and economic evaluation
Li H, Banerjee S, Dunfield L, Kirby J, Jones M, Hamilton J, Deal C, Hadjiyannakis S, Normandin S, Tsakonas E
Record ID 32008000101
English, French
Authors' objectives:
"Given the high cost of rhGH treatment and the evolving evidence base for its clinical effect in patients with TS, policy makers need evidence to inform reimbursement decisions about rhGH." (executive summary)
Authors' results and conclusions:
Six RCTs and nine observational studies were included, ranging in duration from one to eight years. The included studies showed that rhGH treatment accelerates growth and results in improvement in height. No serious AEs were reported in the included studies. QoL data, derived from two RCTs, were variable, precluding any conclusion about rhGH-s influence on QoL. Base case economic analysis showed that the incremental costeffectiveness ratio (ICER) of rhGH treatment versus no treatment was CAD23,630 per centimetre of final height improvement or CAD243,078 per quality-adjusted life year (QALY) gained.
Authors' recommendations:
Implications for Decision Making
1) Treatment with rhGH has a demonstrated impact on final height but its effect on QoL is uncertain. The available evidence suggests that, compared with patients receiving placebo or no treatment, patients who are treated with rhGH experience accelerated growth and improvement in final height. Treatment appears to be safe with no serious AEs and few, if any, AEs reported. QoL data, reported in two studies, were variable and inconclusive.
2) For the average patient, rhGH is cost effective if a payer is willing to pay more than CAD200,000 for a QALY. However, from an ethics perspective, the provision and funding of rhGH could be supported until those with TS reach the lower end of the normal adult height range.
3) Publicly funding rhGH treatment will require additional investment. If it were assumed that all TS patients aged 10 to 15 years were eligible for rhGH therapy, the corresponding annual budget impact for covering ~400 patients across Canada would be CAD11.3 million. The more likely scenario would be that 40% to 50% of eligible patients would receive treatment, with a proportionate decrease in expenditure.
Authors' methods:
Systematic review
Details
Project Status:
Completed
Year Published:
2007
English language abstract:
An English language summary is available
Publication Type:
Not Assigned
Country:
Canada
MeSH Terms
- Human Growth Hormone
- Turner Syndrome
Contact
Organisation Name:
Canadian Agency for Drugs and Technologies in Health
Contact Address:
600-865 Carling Avenue, Ottawa, ON K1S 5S8 Canada. Tel: +1 613 226 2553; Fax: +1 613 226 5392;
Contact Name:
requests@cadth.ca
Contact Email:
requests@cadth.ca
Copyright:
Canadian Agency for Drugs and Technologies in Health (CADTH)
This is a bibliographic record of a published health technology assessment from a member of INAHTA or other HTA producer. No evaluation of the quality of this assessment has been made for the HTA database.